ReviewGene therapy2006
Gene therapy progress and prospects: the eye.
Review in Gene therapy, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 66 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
66 citing papers in PubMed, 1 synthesis or guideline pooled it, 144 citations in OpenAlex.
- Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays.International journal of molecular sciences · 2020Pooled it
- Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026Review
- A multistage cost-effective strategy for the molecular diagnosis of unexplained vision loss patients: practice in inherited ocular fundus disease.Molecular genetics and genomics : MGG · 2026Article
- A Practical Approach to Retinal Dystrophies.Advances in experimental medicine and biology · 2025Review
- The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease.International journal of molecular sciences · 2021Review
- Gene therapy in retinal diseases: A review.Indian journal of ophthalmology · 2021Review
- Retinal gene therapy: an eye-opener of the 21st century.Gene therapy · 2021Article
- Biological drug therapy for ocular angiogenesis: Anti-VEGF agents and novel strategies based on nanotechnology.Pharmacology research & perspectives · 2021Review
- Development of a Corneal Bioluminescence Mouse for Real-Time In Vivo Evaluation of Gene Therapies.Translational vision science & technology · 2020Article
- Stem cell therapies for retinal diseases: from bench to bedside.Journal of molecular medicine (Berlin, Germany) · 2020Review
- Peripheral blood-derived monocytes show neuronal properties and integration in immune-deficient rd1 mouse model upon phenotypic differentiation and induction with retinal growth factors.Stem cell research & therapy · 2020Article
- Stable Retinoid Analogue Targeted Dual pH-Sensitive Smart Lipid ECO/pDNA Nanoparticles for Specific Gene Delivery in the Retinal Pigment Epithelium.ACS applied bio materials · 2020Article
- Gene Therapy in Retinal Dystrophies.International journal of molecular sciences · 2019Review
- Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration.Frontiers in neuroscience · 2019Article
- Principles of pharmacology in the eye.British journal of pharmacology · 2017Review
- Insights from Genetic Model Systems of Retinal Degeneration: Role of Epsins in Retinal Angiogenesis and VEGFR2 Signaling.Journal of nature and science · 2017Article
- Article
- Self-Assembly of a Multifunctional Lipid With Core-Shell Dendrimer DNA Nanoparticles Enhanced Efficient Gene Delivery at Low Charge Ratios into RPE Cells.Macromolecular bioscience · 2015Article
- Gene therapy for ocular diseases meditated by ultrasound and microbubbles (Review).Molecular medicine reports · 2015Review
- Promising and delivering gene therapies for vision loss.Vision research · 2015Review
6 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The eye has unique advantages as a target organ for gene therapy of both inherited and acquired ocular disorders and offers a valuable model system for gene therapy. The eye is readily accessible to phenotypic examination and investigation of therapeutic effects in vivo by fundus imaging and electrophysiological techniques. Considerable progress has been made in the development of gene replacement therapies for retinal degenerations resulting from gene defects in photoreceptor cells (rds, RPGRIP, RS-1) and in retinal pigment epithelial cells (MerTK, RPE65, OA1) using recombinant adeno-associated virus and lentivirus-based vectors. Gene therapy also offers a potentially powerful approach to the treatment of complex acquired disorders such as those involving angiogenesis, inflammation and degeneration, by the targeted sustained intraocular delivery of therapeutic proteins. Proposals for clinical trials of gene therapy for early-onset retinal degeneration owing to defects in the gene encoding the visual cycle protein RPE65 have recently received ethical approval.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.